Gene Editing Technique Successful with Nanoparticles

Yale researchers have successfully corrected, the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. To correct the mutation, the multidisciplinary team used synthetic molecules similar to DNA — called peptide nucleic acids, or PNAs — as well as donor DNA, to edit the genetic defect. The researchers also developed a method of delivering the PNA/DNA via microscopic nanoparticles.

The researchers also developed a method of delivering the PNA/DNA via microscopic nanoparticles. These tiny particles, which are billionths of a meter in diameter, are specifically designed to penetrate targeted cells.

In both human airway cells and mouse nasal cells, the researchers observed corrections in the targeted genes. “The percentage of cells in humans and in mice that we were able to edit was higher than has been previously reported in gene editing technology,” said researchers. They also observed that the therapy had minimal off target, or unintended, effects on treated cells.

In both human airway cells and mouse nasal cells, the researchers observed corrections in the targeted genes.